The Charcot-Marie-Tooth Association (CMTA) is excited to share that our Alliance Partner, NMD Pharma, has received clearance from the US Food and Drug Administration (FDA) to initiate a Phase II clinical trial for their investigational drug, NMD670, in patients with Charcot-Marie-Tooth disease (CMT). This trial, named SYNAPSE-CMT, marks a significant milestone in the journey towards finding effective treatments for CMT.

What is NMD670?

According to NMD Pharma, its muscle-targeted oral drug treatment, NMD670, is a first-in-class small molecule inhibitor of the ClC-1 chloride ion channel specific to skeletal muscles. By inhibiting ClC-1, NMD670 aims to improve neuromuscular transmission to enhance skeletal muscle activation and improve muscle function. This innovative approach is particularly promising for people with CMT, where muscle weakness and atrophy are predominant symptoms.

About the SYNAPSE-CMT Trial

The Phase II SYNAPSE-CMT trial is a randomized, double-blinded, placebo-controlled study that will evaluate the efficacy and safety of NMD670 in approximately 84 adult patients with either demyelinating or axonal CMT. The trial will involve twice daily oral administration of NMD670 and will be conducted at multiple clinical sites across the US and Europe. NMD Pharma expects enrollment to begin soon.

Background and Significance

In June 2023, NMD Pharma presented findings from their ESTABLISH study, an international observational study that revealed neuromuscular junction transmission deficits as a characteristic of CMT. This study demonstrated that patients with CMT exhibit varying degrees of neuromuscular transmission weakness, which correlates with disease severity. These findings underscore the potential of NMD670 to address a critical aspect of CMT that was previously unknown.

Thomas Holm Pedersen, CEO of NMD Pharma, stated, “Charcot-Marie-Tooth is a highly debilitating condition with no cure or approved treatments. The results from our ESTABLISH study highlight the potential of NMD670 to address neuromuscular junction deficits in CMT, offering new hope for patients. We are eager to advance this trial and move closer to providing a much-needed therapeutic option for the CMT community.”

CMTA’s Support and Outlook

Sue Bruhn, PhD, CEO of CMTA, expressed the organization’s enthusiasm: “We are extremely proud to have NMD Pharma as an Alliance Partner. The FDA’s IND clearance for the NMD670 Phase 2 trial in CMT marks a significant step forward in our mission to bring effective treatments to patients, and we are thrilled that our investment in clinical trial readiness will accelerate progress by helping recruit participants through our Patients as Partners in Research platform.”

CMTA remains committed to supporting NMD Pharma and closely following the progress of the SYNAPSE-CMT trial. We are hopeful that our partnership and the ongoing research will lead to significant advancements in treating CMT and improving the lives of those affected by this condition.

Register with Patients as Partners in Research Today

CMTA will soon contact Patients as Partners in Research registrants who match the study criteria for NMD Pharma’s SYNAPSE-CMT trial. CMTA will contact only those who previously consented to receive research opportunity notifications.

Not yet registered with Patients as Partners in Research but wish to participate in the SYNAPSE-CMT trial? Click the button below to register today! Registering today ensures you are the first in line when NMD is ready to enroll community members in this study. Registering also ensures you are updated on all the latest news and developments in CMT research. Registration is free and secure, and all who have CMT are invited to register.

Create Your Patients As Partners In Research Profile Today

Published: June 18, 2024